FDA Approves First Pediatric Indication for Blockbuster Eylea


Eylea now also treats retinopathy of prematurity, a leading cause of childhood blindness worldwide.

The FDA approved the first pediatric indication for Regeneron’s Eylea (aflibercept) to treat preterm infants with retinopathy of prematurity (ROP). Retinopathy of prematurity is a leading cause of childhood blindness worldwide. Between 1,100 to 1,500 infants in the United States annually develop ROP that is severe enough to require medical treatment. The rare eye disease often impacts infants who are born before 31 weeks of pregnancy have been completed or who weigh less than 3.3 pounds at birth.

George D. Yancopoulos, M.D., Ph.D.

George D. Yancopoulos, M.D., Ph.D.

Until now, the only FDA-approved treatment in common use was laser photocoagulation, “a complex and lengthy procedure that permanently ablates retina tissue and is stressful not only for infant patients but also the family navigating a delicate time after a preterm birth,” saidGeorge D. Yancopoulos, M.D., Ph.D., president and chief scientific officer of Regeneron, and a principal inventor of Eylea.

Bayer and Regeneron are collaborating on the global development of Eylea, and Regeneron maintains exclusive rights in the United States.

Eylea is currently available to treat patients with age-related macular degeneration, diabetic macular edema and diabetic retinopathy. It is a VEGF inhibitor that is injected into the eye. It is designed to block the growth of new blood vessels and decrease the ability of fluid to pass through blood vessels in the eye.

The blockbuster Eylea, which realized sales of $6.26 billion in 2022, is now indicated to treat five retinal conditions caused by ocular angiogenesis, Regeneron said. While Regeneron has not released the wholesale acquisition cost (WAC) for the infant dose, the current WAC per prefilled syringe or single-dose vial 2mg/0.05mL is $1,850.

The FDA granted a Pediatric Exclusivity Determination in October, extending the period of U.S. market exclusivity for Eylea by an additional six months through May 17, 2024.

“With no existing FDA approved guidance for the treatment of retinopathy of prematurity with anti-VEGF therapies, there was a significant need for research to understand how best to treat the disease in a manner that puts patient safety first and preserves vision for a lifetime,” said Jeff Todd, CEO of Prevent Blindness. “Regeneron’s trials investigating Eylea in retinopathy of prematurity have advanced our understanding of how to treat this disease and provided a needed evidence-based treatment option to potentially help preterm infants preserve their vision.”

The FDA approval is supported by data from two randomized global phase 3 trials — FIREFLEYE and BUTTERFLEYE — investigating EYLEA 0.4 mg versus laser photocoagulation (laser) in infants with ROP. In both trials, approximately 80% of EYLEA-treated infants achieved an absence of both active ROP and unfavorable structural outcomes at 52 weeks of age, which is better than would have been expected without treatment, Regeneron said.

No new safety signals were observed in either trial. Comparing Eylea with laser, ocular adverse events (AEs) among patients occurred in 39% versus 37% in the FIREFLEYE study and 18% versus 26% in BUTTERFLEYE trial, with serious ocular AEs occurring in 8% for both groups in FIREFLEYE and 6.5% versus 11% in BUTTERFLEYE.

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